PALO ALTO, Calif.--(BUSINESS WIRE)--LifeMax Laboratories, Inc. (“LifeMax”), a private company focused on treating rare diseases with few or no therapeutic options, today announced that the European Commission granted orphan drug designation to LM-030, an investigational therapy licensed from Novartis and ready to enter into a pivotal clinical trial for the treatment of Netherton Syndrome. LM-030 previously received orphan drug designation in June and rare pediatric disease designation in July 2019 from the FDA.
“We are very pleased to have received orphan drug designation for LM-030 from the European Commission, which, together with orphan drug designation and rare pediatric disease designation from FDA, serves to position us well for a global regulatory development pathway. Netherton Syndrome is a severe genetic disorder with neonatal onset that can be life-threatening. Receiving orphan drug designation represents another significant step in the development of LM-030 for this devastating disease,” said Larry Hsu, LifeMax’s Co-founder and CEO, an industry veteran who previously founded and built Impax Laboratories into a publicly traded multi-billion dollar company.
Orphan drug designation is granted by the European Commission for a medicine that is intended to treat and offers significant benefit to a life-threatening or chronically debilitating disease affecting less than 5 in 10,000 people in the European Union. Benefits that come with an orphan drug designation include, but are not limited to, reduced or exempted fees and ten-year market exclusivity.
About Netherton Syndrome
Netherton Syndrome is a severe autosomal recessive disease characterized by congenital erythroderma, “bamboo hair” and immune system abnormalities. It can be life-threatening in pediatrics due to an impaired skin barrier that leads to severe dehydration, hypernatremia, hypothermia, gross weight loss and sepsis. Failure to thrive is common in childhood as a result of chronic erythroderma, persistent cutaneous infection, malnutrition and metabolic disorders. The severity of the skin abnormality in older patients can fluctuate over time. Most Netherton Syndrome patients are also inflicted with immune system-related disorders such as food allergies and asthma.
Though the exact prevalence of Netherton Syndrome is not well documented, it has a reported prevalence of 1-9 per 1,000,000.
About LifeMax Laboratories Inc.
LifeMax Laboratories, Inc. (www.lifemaxlabs.com), a wholly-owned subsidiary of LifeMax Healthcare International Corporation (www.lifemaxhealthcare.com), is a clinical-stage biotech company focused on treating rare diseases with few or no therapeutic options. LifeMax is applying its expertise and in-depth experience with drug development and commercialization to advance its well-balanced portfolio of products addressing critical unmet medical needs.